Findings from a Phase 1/2a clinical trial of its Multi-Characteristic Opsin (MCO) gene therapy for people blinded by retinitis pigmentosa (RP) were highlighted in two presentations during the 2021 Annual Meeting of the American Society of Retina Specialists (ASRS).

The presentations were made Dr. Sai Chavala, MD, a professor at TCU-UNTHSC Medical School. Dr. Chavala’s presentation titled, “Intravitreal AAV2 Optogenetic Vision Restoration in Retinal Degenerative Patients with ABCA4 Mutation,” on Sunday, October 10, 2:46 pm -2:50 pm, during an In the Pipeline session exploring new therapies.

Dr. Chavala detailed findings from four RP patients with ABCA4 mutation from the Phase1/2a trial who showed overall improvement in vision after 52 weeks of receiving a single intravitreal injection of MCO-010.

Dr. Chavala discussed data from all 11 RP patients in the Phase1/2a trial in a Video on Demand, titled “Safety and Efficacy of Intravitreal Optogenetic Gene Therapy for Advanced Retinitis Pigmentosa,” as part of the ASRS Virtual Annual Meeting Program. Results showed the therapy was well-tolerated and all patients experienced improvement in vision following MCO-010 injection.

MCO-010 uses a proprietary AAV2 vector to deliver MCO genes into retinal cells where they express polychromatic opsins sensitive to ambient light for vision restoration in different color environments. The therapy focuses on disease phenotype, enabling treatment of retinal degenerative diseases regardless of genetic mutations.

Results of a randomized, double-masked Phase 2b multi-center trial (NCT04945772) of MCO-010s are expected in Q4 2022. MCO-010 has orphan drug designations from the FDA for RP and Stargardt disease, which are known to be associated with ABCA4 mutation.